The company, which has raised a total of $42m, intends to use the funds to expand development of its lead product to treat NPC.

Led by Ben Machielse, President and Chief Executive Officer, Vtesse is working collaboratively with the NIH, other academic centers, parents, and patient advocacy groups, to advance a pivotal clinical study of VTS-270, a well-characterized mixture of HPbCD with a specific compositional fingerprint to treat NPC, a progressive, irreversible, chronically debilitating – and lethal – genetic disease, and to conduct pre-clinical discovery and development of other novel drugs for NPC and other lysosomal storage diseases (LSDs).

In January 2016, the company announced that the U.S. Food & Drug Administration (FDA) had granted Breakthrough Therapy designation status for VTS-270 for treatment of NPC. Both the FDA and the European Medicines Agency (EMA) had previously granted Orphan Drug status to VTS-270.